UCB presents final open-label extension (fenfluramine) data at International Child Neurology Congress (ICNC) Annual Meeting.

As of January 2023, 375 patients were enrolled in the OLE; 92 were 2 to less than 6 years old and received >= 1 dose of fenfluramine.

The data demonstrated that treatment with fenfluramine for up to 3 years in people living with Dravet syndrome was generally well tolerated, with no new or unexpected safety signals identified, and patients experiencing clinically significant, durable reductions in monthly convulsive seizure frequency (MCSF).

In the modified intent-to-treat (mITT) population (n=324), the median percent MCSF change from baseline over the entire OLE was -66.8%, P<0.001. in the mitt population of patients 2 to less than 6 years old (n="85)," median mcsf change from baseline during the overall ole was -74.2%, p><0.001. in addition, global functioning scores – as measured by the clinical global impression (cgi) scale - were “much improved” or “very much improved” from fenfluramine treatment by 61.4% and 62.5% of caregivers and investigators, respectively. in patients under 6 years of age, clinically meaningful improvement was reported by 69.0% and 66.7% of caregivers and investigators, respectively.>

No new or unexpected treatment-emergent adverse events were observed in the studies, and there were no cases of valvular heart disease or pulmonary arterial hypertension. The most common (occurring in greater than 15% of patients during the OLE) treatment-emergent adverse events were pyrexia, nasopharyngitis, decreased appetite, decreased blood glucose diarrhea, upper respiratory tract infection and seizure.

“The final analyses from the open-label extension (OLE) studies being presented at this year’s ICNC meeting reinforce the long-term treatment potential and efficacy of fenfluramine for up to three years, showing profound, durable reductions in median monthly convulsive seizure frequency in people living with Dravet syndrome,” commented Dr Elaine C. Wirrell, principal investigator. “The ultimate goals are reducing seizure frequency for the patient and maximizing developmental potential and quality of life.”

“The data being presented at ICNC highlight the seizure, non-seizure, and improved survival outcomes of fenfluramine as well as our unwavering commitment to transforming care and improving the quality of life for people living with epilepsies,” said Konrad Werhahn, Global Head of Medical Affairs, Epilepsy & Rare Syndromes, UCB. “Hosting this essential gathering on African soil marks a momentous occasion. Approximately 25 million people in Africa are living with epilepsy, and yet more needs to be done to improve patient care and support.Everything we do is centered around providing treatment options to people and families across the world living with seizure disorders and helping them to maximize their life opportunities.”